Drug updated on 4/17/2024
| Dosage Form | Injection (intravenous; 200 units/vial) |
| Drug Class | Hydrolytic lysosomal enzymes |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of type 1 Gaucher disease in patients 4 years and older with a confirmed diagnosis.
Summary
- Taliglucerase alfa (Elelyso) is used for the treatment of type 1 Gaucher disease in patients aged 4 years and older with a confirmed diagnosis. It is an enzyme replacement therapy that has been found effective in both naïve and experienced patients.
- The information was derived from a systematic review/meta-analysis study, which compared Elelyso to other drugs for treating Gaucher disease but did not provide specific comparative effectiveness or safety data.
- Other enzyme replacement therapies mentioned include Imiglucerase and Velaglucerase alfa, which are also suggested to be effective treatments for Gaucher disease. However, no direct comparison between these drugs and Taliglucerase alfa was provided.
- Substrate Reduction Therapy (SRT) Treatments such as Miglustat showed no significant improvement in blood outcomes for naïve patients, while Eliglustat resulted in stable outcome values mainly assessed in experienced patients, implying some effectiveness.
- No specific details regarding the safety profiles of Taliglucerase alfa were provided by the study; it focused more on drug efficacy than on side effects or tolerability issues.
- This document highlights effectiveness across patient types—those who have never received treatment before (naïve), as well as those who have previously undergone treatment (experienced); there is a lack of subgroup analysis based on age, severity of illness, or other demographic factors.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Elelyso (taliglucerase alfa) Prescribing Information. | 2023 | Pfizer Inc., New York, NY |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| A systematic review and meta-analyses of longitudinal studies on drug treatments for Gaucher disease. | 2023 | Annals of Pharmacotherapy |
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Management goals of type 1 Gaucher disease in South Africa: an expert Delphi consensus document on good clinical practice. | 2023 | PLoS One |
| Recommendations for oral treatment for adult patients with type 1 Gaucher disease. | 2022 | Revista Clínica Española |